Navigation Links
Erasing a genetic mutation
Date:3/30/2014

CAMBRIDGE, MA -- Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation.

The findings, described in the March 30 issue of Nature Biotechnology, offer the first evidence that this gene-editing technique, known as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers an easy way to snip out mutated DNA and replace it with the correct sequence, holds potential for treating many genetic disorders, according to the research team.

"What's exciting about this approach is that we can actually correct a defective gene in a living adult animal," says Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering at MIT, a member of the Koch Institute for Integrative Cancer Research, and the senior author of the paper.

The recently developed CRISPR system relies on cellular machinery that bacteria use to defend themselves from viral infection. Researchers have copied this cellular system to create gene-editing complexes that include a DNA-cutting enzyme called Cas9 bound to a short RNA guide strand that is programmed to bind to a specific genome sequence, telling Cas9 where to make its cut.

At the same time, the researchers also deliver a DNA template strand. When the cell repairs the damage produced by Cas9, it copies from the template, introducing new genetic material into the genome. Scientists envision that this kind of genome editing could one day help treat diseases such as hemophilia, Huntington's disease, and others that are caused by single mutations.

Scientists have developed other gene-editing systems based on DNA-slicing enzymes, also known as nucleases, but those complexes can be expensive and difficult to assemble.

"The CRISPR system is very easy to configure and customize," says Anderson, who is also a member of MIT's Institute for Medical Engineering an
'/>"/>

Contact: Sarah McDonnell
s_mcd@mit.edu
617-253-8923
Massachusetts Institute of Technology
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. Genetic mutations warn of skin cancer risk
2. Drilling into the trends in genetics and epigenetics of aging and longevity
3. Faster genetic testing method will likely transform care for patients with breast cancer
4. Genetics can explain why infections can trigger rheumatoid arthritis
5. Certain genetic variants may put bladder cancer patients at increased risk of recurrence
6. MRI reveals genetic activity
7. UT Southwestern ob/gyn researchers studying genetic factors in premature births
8. Researchers issue state-of-the-state on genetic-based testing & treatment for breast cancer
9. TGen-led study discovers genetic cause of rare type of ovarian cancer
10. Genetic testing may help select women with ER+ breast cancer for extended hormone therapy
11. Genetic test could improve colon cancer screening
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/11/2014)... Minn. , Dec. 10, 2014  Data ... physiologic monitoring, has released a new series of ... of preclinical toxicology researchers. M series, part of ... toxicologists collect the best possible physiologic data when ... Adding functional endpoints to toxicology studies has ...
(Date:12/10/2014)... , Dec. 9, 2014  Valencell, a leader in ... seeing a staggering demand from its licensees for highly ... revealed, is not solely coming from fitness and health ... "A wearable is only as useful as ... is the ultimate driver in long-term mass consumer adoption ...
(Date:12/10/2014)... CIE San Diego has launched an innovative ... tissue that enhances care coordination among social service and ... information; earned a second $1 million grant from the ... aging in community and; will be recognized as a ... 4-6p. CIE San Diego today announced ...
Breaking Biology News(10 mins):New telemetry implants expected to change how large animal toxicology studies are conducted 2Wearable Technology Products Demand Highly Accurate Biometric Technology 2Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 2Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 3Community Information Exchange San Diego Launches Pilot, Secures $1 Million Expansion Grant, and Becomes a "Live Well San Diego" Partner 4
... ANN ARBOR, Mich.---In the quest for new approaches to ... on or off at will, directly intervening in ailments such ... turn on and off as they should. Scientists at ... Berkeley have taken a step forward on that route by ...
... University of Delaware have discovered a new reason why the tall, ... invasive plants in the United States. The UD research team ... to snuff out its victims, and the poison becomes even more ... study, which is published in the June issue of the scientific ...
... an international team of scientists led by the University of ... the cave of El Mirn. Since then they have focused ... of small vertebrates that lived in the Cantabrian region over ... The richness, great diversity and good conservation status of the ...
Cached Biology News:Small molecules mimic natural gene regulators 2Changing climate likely to make 'super weed' even more powerful 2Cantabrian cornice has experienced seven cooling and warming phases over past 41,000 years 2
(Date:12/22/2014)... 2014 Fertility Associates of Memphis has ... USA , a network of more than 20 contributing ... patients have fast access to a national pool of ... . Fertility Associates of Memphis is the only fertility ... option. , Dr. William Kutteh , director of ...
(Date:12/19/2014)... BioPlus Specialty Pharmacy (BioPlus), one of the nation’s ... Pharm.D. to the newly created position of Vice President ... Dr. Maroulis will continue to manage the entire specialty ... pharmacies as the company expands. Dr. Maroulis has been ... has served in many different capacities in the pharmacy, ...
(Date:12/19/2014)... , Dec. 19, 2014 Bina Technologies, Inc. (Bina, ... today that they have been acquired by Roche (SIX: ... a privately held company that provides a big data ... sequencing (NGS) data for the academic and translational research ... Unit, and will continue to focus on development of ...
(Date:12/19/2014)... Altamonte Springs, FL (PRWEB) December 18, 2014 ... one of the nation’s leading specialty pharmacies, has ... who serve as clinical pharmacy consultants for prescriber ... disease therapy expertise. , “We have a ... disease therapies. For example, those specializing in hepatitis ...
Breaking Biology Technology:Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 2Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 3BioPlus Specialty Pharmacy Promotes Dr. Nick Maroulis to Vice President of Specialty Pharmacy Services 2Bina Technologies is acquired by Roche. 2Bina Technologies is acquired by Roche. 3BioPlus Specialty Pharmacy Expands ‘Regional Pharmaceutical Care Liaison’ Team 2BioPlus Specialty Pharmacy Expands ‘Regional Pharmaceutical Care Liaison’ Team 3
... key engineering challenges to building a clean, efficient, hydrogen-powered ... enough raw hydrogen for a reasonable driving range would ... extremely low temperatures for liquid hydrogen. In a new ... Technologys Center for Neutron Research (NCNR) have demonstrated that ...
... 2008 With their ability to extend,product life, excipients ... strategy during lean times, and this portends a robust ... major life sciences market research,publisher. There are about 1200 ... This $4.6 billion market has witnessed healthy growth,in the ...
... April 2, 2008 PAREXEL International,Corporation (Nasdaq: ... services,organization, today announced that Larry Ereshefsky, Pharm.D., ... appointed as Vice Presidents,of Clinical Pharmacology Consulting ... These leading experts advise clients on,early phase ...
Cached Biology Technology:More solid than solid: A potential hydrogen-storage compound 2Excipients Help Pharma Extend Product Life, Profits 2PAREXEL Appoints Leading Central Nervous System Experts to Its Global Clinical Pharmacology Business 2PAREXEL Appoints Leading Central Nervous System Experts to Its Global Clinical Pharmacology Business 3PAREXEL Appoints Leading Central Nervous System Experts to Its Global Clinical Pharmacology Business 4PAREXEL Appoints Leading Central Nervous System Experts to Its Global Clinical Pharmacology Business 5
Edited by D.M. Gersten (1996) Provides comprehensive descriptions of the techniques available for analytical gel electrophoresis of proteins and offers clear step-by-step protocols for all of the m...
...
All serum is collected from U.S.D.A. fryers and broilers. Pricing: $15/unit for 1 - 10 units; $12/unit for 11 - 50 units...
RAT ANTI MOUSE TCR V BETA 7:RPE Immunogen: T-cell lymphoma TK1...
Biology Products: